Stem cell clinical trials to treat eye diseases begin at UCLA

By Daniela Hernandez, Los Angeles Times/For the Booster Shots blog

July 14, 2011 12 AM PT

After more than 20 years of research, doctors at UCLA’s Jules Stein Eye Institute have begun treating the first patients in clinical trials for two progressive eye diseases that cause blindness: dry age-related macular degeration and Stargardt’s macular dystrophy.

For the Record, 12:27 p.m. July 26: An earlier version of this post had an incorrect age for the patient with Stargardt’s macular degeneration. She is actually older, according to UCLA officials, though they declined to give her exact age. Also, in the original post Stargardt’s was misspelled once as Stardgart’s.

The patients were given an injection of specialized eye cells that were derived from embryonic stem cells. Dr. Steven Schwartz, who is leading the trial at UCLA, performed both stem cell transplant surgeries Tuesday. The two patients are said to be recovering without complications.

According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technologies Inc., which developed the cells and is sponsoring the trials, “you could feel the excitement in the air and that history was being made.”

Surgery began for the first of two patients, a 77-year old woman with dry macular degeneration, around 9:30 a.m. Tuesday and took about half an hour. In the afternoon, a woman with Stargardt’s macular dystrophy underwent the same procedure. Both patients are legally blind.

Doctors will monitor these two patients over the coming weeks. Another set of surgeries is scheduled to start in August.

Schwartz explained how these trials will help patients and what they mean for regenerative medicine. His comments were edited for space and clarity.

What are dry age-related macular degeneration and Stargardt’s macular dystrophy?

Twenty percent of age-related macular degeneration is wet macular degeneration and it is treatable. The other 80% of people have an untreatable, progressive visual loss leading to legal blindness called dry, or atrophic, macular degeneration.

The retinal cells, the rods and cones, and the underlying retinal pigment epithelium atrophy. As they atrophy, there is a long period of time when they are compromised and then they’re gone.

Stargardt’s is a genetic disorder and it strikes earlier in life. Patients start to notice visual changes as early as their teens and as late as their forties. There are a number of known genetic abnormalities in the photoreceptors that are toxic over time.

In this trial, what cells in the eye are you replacing with stem cells?

Advanced Cell Technologies Inc. has been able to take [embryonic] stem cells and differentiate them into highly functional retinal pigment epithelium that do everything they’re supposed to do. Our strategy of giving the eye brand-new, ready-to-go retinal pigment epithelium is designed for areas that are compromised, not for the areas that are gone. So we need to catch it early enough for this treatment to work.

Will the patients regain vision?

The patients’ central vision is already gone. Not rescuable. So the patients we’re enrolling in this trial know they will not be getting their central vision back.

If not to restore vision, what is the goal?

This is a safety trial. It’s not designed to improve vision. It may; and if we see a signal, that would be great news and we’re hoping we will. It’s plausible biologically, but that’s not what we’re looking for.

What results are you hoping to see?

I hope what happens is that we find this is safe and that we can optimize the dosing, and that allows us to move into eyes that are earlier in disease. That could have a real visual upside.

How long is the surgery?

Under an hour. It’s an outpatient procedure done with local anesthesia. It’s a surgery that we’ve done before – not with the injection of these stem cells, but we’ve accessed the eye before, and that’s one of the things that I’ve had a lot to do with surgically.

What does this trial mean for medicine?

We’re super-privileged to be taking this first step. It’s the unknown. These patients are doing a service for mankind. It’s inspirational.